Antonio-Aguirre Bani, Arevalo J Fernando
Wilmer Eye Institute, Johns Hopkins School of Medicine, 600 N Wolfe St; Maumenee 713, Baltimore, MD, 21287, USA.
Int J Retina Vitreous. 2023 Nov 20;9(1):72. doi: 10.1186/s40942-023-00493-6.
Geographic atrophy (GA) is a progressive degenerative disease that significantly contributes to visual impairment in individuals aged 50 years and older. The development of GA is influenced by various modifiable and non-modifiable risk factors, including age, smoking, and specific genetic variants, particularly those related to the complement system regulators. Given the multifactorial and complex nature of GA, several treatment approaches have been explored, such as complement inhibition, gene therapy, and cell therapy. The recent approval by the Food and Drug Administration of pegcetacoplan, a complement C3 inhibitor, marks a significant breakthrough as the first approved treatment for GA. Furthermore, numerous interventions are currently in phase II or III trials, alongside this groundbreaking development. In light of these advancements, this review provides a comprehensive overview of GA, encompassing risk factors, prevalence, genetic associations, and imaging characteristics. Additionally, it delves into the current landscape of GA treatment, emphasizing the latest progress and future considerations. The goal of starting this discussion is to ultimately identify the most suitable candidates for each therapy, highlight the importance of tailoring treatments to individual cases, and continue monitoring the long-term implications of these emerging interventions.
地图样萎缩(GA)是一种进行性退行性疾病,对50岁及以上人群的视力损害有显著影响。GA的发展受多种可改变和不可改变的风险因素影响,包括年龄、吸烟和特定的基因变异,尤其是那些与补体系统调节因子相关的基因变异。鉴于GA的多因素和复杂性,人们探索了多种治疗方法,如补体抑制、基因治疗和细胞治疗。美国食品药品监督管理局最近批准了补体C3抑制剂培西加可普坦,这是GA首个获批的治疗方法,标志着重大突破。此外,在此突破性进展的同时,众多干预措施目前正处于II期或III期试验阶段。鉴于这些进展,本综述全面概述了GA,包括风险因素、患病率、基因关联和影像学特征。此外,还深入探讨了GA治疗的现状,强调了最新进展和未来考虑因素。展开此次讨论的目的是最终确定每种疗法最适合的患者,突出针对个体病例量身定制治疗的重要性,并持续监测这些新兴干预措施的长期影响。